Research finds way for 40pc thalassaemic children to lead normal life

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KARACHI: Around 40 per cent of thalassaemic children have finally found a panacea for their ailment in the shape of a Rs10 capsule which if used on a daily basis can offer them independence from blood transfusion, said an eminent haematologist on Friday.

“Our medicine is making wonders, putting lives on a normal footing and saving huge money and blood that has been spent on the efforts to save thalassaemia patients,” claimed Dr Saqib Hussain Ansari, the haematologist who has been running the Omair Sana Foundation charity for thalassaemic children for the past several years.

Take a look: ‘Rs10bn is spent annually on thalassaemia treatment’

He was speaking at a seminar on thalassaemia held at the foundation office. The event was part of several programmes organised by the medical fraternity and organisations to mark International Thalassaemia Day.

Thalassaemia is a hereditary disease in which children need fresh blood from donors after short intervals for their entire life, as their bodies are unable to produce haemoglobin, a vital part of blood to stay alive and healthy.

Dr Ansari said that the life of thalassaemic patients could be improved significantly with the use of this medicine, hydroxyurea (HU), which had been tested repeatedly with a success rate of 40pc. “We are highly relieved to announce the results of our latest tests on 3,000 children suffering from thalassaemia, which show around 40pc of children respond to the medicine positively and those young patients have now no need to transfuse blood every now and then,” he claimed.

The haematologist said the medical tests of the medicine were launched in 2003 with 23 patients. It was later expanded to 146 thalassaemic children when the results showed positive improvement in more than 40pc children. Now, he said, the ratio of the success remained around 40pc when HU was tested on 3,000 children with thalassaemia major.

Of the remaining 60pc children, said the expert, 30pc responded partially, with the result that the duration of their blood transfusion increased doubly. But the remaining 30pc children remained unreceptive to the medicine, he added.

“It is because of genetics,” he explained, “we have identified certain genes in thalassaemic children that are chiefly responsible for the response to HU — completely or partially — or not, yet the results show we can give normal life to 40pc of our thalassaemic children and reduce pain of transfusion to half of the remaining.”

Dr Ansari said he found during his research work that the use of HU ended the dependence of blood transfusion in 40 per cent of 146 children suffering from blood disorders due to six of 17 genetic disorders that resulted in thalassaemia major. The drug reduced the dependence of 39 per cent children on blood transfusion to half, while it remained ineffective in remaining 20 to 21 per cent patients, he said.

The research work, initiated in 2003, was carried out at the National Institute of Blood Diseases (NIBD) where children with genetic blood disorders are brought for treatment.

Dr Ansari said he had got two papers on his research published in US Journal of Paediatric Haematology and Oncology in 2007 and 2011.

After more than 10 years of hectic research, he said, it was discovered that the drug was effective in children suffering from thalassaemia due to six of the total 17 genetic disorders that caused thalassaemia and some other genetic blood disorders.

“I have completed my PhD on the same topic and my research revealed that use of this drug can end or minimize dependence of blood in most of the children,” he said.

He said the use of HU did not result in increasing the size of spleen or livers of affected patients.

According to him, hydroxyurea is an FDA-approved drug for patients suffering from sickle cell anaemia and is being used in the US for the past 20 years. He said that the use of this drug also produced very encouraging results in Iran and India.

He said that the anti-neoplastic drug improved the health of thalassaemic children, who were given the drug, as their weight and appetite improved while their interest in playing and other healthy activities also enhanced.

His research showed that in Pakistan, 37 per cent patients were found with both alpha and beta thalassaemia disorders.

“Every year, 6,000 patients with beta thalassaemia are born in Pakistan, while there are around 9.8 million carriers of this disease in the country,” he said.

“If the new drug is applied to thalassaemia children,” said the haematologist, “Rs12 billion and 500,000 blood bags can be saved annually.”

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